Phase I/II study shows safety and efficacy of VB-111 in patients with rGBM

VBL Therapeutics, a clinical-stage biotechnology company committed to the development of novel treatments for immune-inflammatory diseases and cancer, today announced clinical data demonstrating the utility of VB-111's in targeted cancer treatment. The company presented results from Phase I/II clinical study in patients with recurrent glioblastoma at the annual meeting of the American Society of Clinical Oncology (ASCO) in Chicago. Data from the trial evaluating the effect of VB-111 on 28 patients with recurrent Glioblastoma Multiforme (rGBM) demonstrate that VB-111 was safe and well tolerated with repeat doses of up to 1x10¹³ VPs. Tumor responses and significant attenuation of tumor growth rate were seen. Overall survival was about 3 months longer compared to historical data in rGBM with the standard of care with anti-angiogenic agents.

“We were pleased to present our promising results at the ASCO conference”

The poster (#TPS2102) entitled "Phase I/II dose-escalation study of VB-111, an antiangiogenic gene therapy, in patients with recurrent glioblastoma multiforme," by Brenner et al., was presented on June 1^st, 2013, and its abstract is now available on the ASCO annual meeting website.

VB-111 is a novel anti-angiogenic gene-therapy tool that targets endothelial cells in the tumor vasculature. Based on a non-replicating adenoviral vector, it harbors a uniquely modified pre-proendothelin promoter (PPE-1-3x) which regulates transcription of a Fas-Chimera transgene. The proprietary promoter specifically targets expression of the Fas-Chimera transgene to angiogenic tumor blood vessels, leading to their apoptosis, with no harm to normal vasculature and non-cancerous tissues in the body. VB-111 is the first agent based on transcriptional targeting of tumor endothelium to be assessed in a clinical trial.

Twenty eight patients with rGBM aged 26 - 74 years, enrolled the trial between Dec.2010 - Aug.2012 in three US medical centers: the Dana-Farber Cancer Institute, Duke University Medical Center and the University Of Texas Health Science Center. Patients received up to 8 repeat doses of VB-111. The median overall survival was 360 days for patients receiving at least one dose of 1x10¹³ VPs (high dose). Progression free survival was 87 vs 55 days for patients who received high dose vs. lower doses, respectively>

"We were pleased to present our promising results at the ASCO conference", said Professor Dror Harats, M.D., chief executive officer of VBL. "The efficacy and unique endothelial-specificity of our VTS^TM platform is evidently translated to humans. Our data demonstrate VB-111's potency as a targeted cancer treatment, which seems to prolong overall survival in rGBM patients. We are confident in the therapeutic potential of VB-111 and continue to advance its clinical trials in gliobastoma multiforme, differentiated thyroid cancer and ovarian cancer".

Phase I data for VB-111 was recently published in the journal of Clinical Cancer Research. The manuscript reported a first in man clinical trial of VB-111, demonstrating its safety and tolerability in patients with advanced metastatic cancer at a single administration of up to 1x10¹³ Viral Particles (VPs). Notably, tumor response and superior overall survival were found in the 1x10¹³ VPs cohort compared to sub-therapeutic doses. The data confirm pre-clinical findings in animal models and validate VB-111's mechanism of action, resulting in a targeted expression of the Fas-Chimera transgene selectively in tumor vasculature.